Astria Therapeutics(ATXS) - 2020 Q1 - Quarterly Report

Drug Development - The company is focused on developing edasalonexent, an oral small molecule for treating Duchenne muscular dystrophy (DMD), which has received orphan drug and fast track designations from the FDA [57]. - The Phase 3 PolarisDMD trial for edasalonexent completed enrollment in September 2019 with 131 boys, exceeding the target of 125 [58]. - Top-line results from the Phase 3 PolarisDMD trial are expected in Q4 2020, with plans to submit a New Drug Application in 2021 [58]. - The company initiated a partnership with Duchenne UK, receiving over $600,000 in funding for a planned Phase 2 trial of edasalonexent in non-ambulatory DMD patients [63]. - The GalaxyDMD trial was launched in March 2019 to provide long-term safety data following the Phase 3 trial [59]. - The company is exploring edasalonexent for other indications, including dysferlinopathy, through a collaboration with the Jain Foundation [64]. - CAT-5571, developed for cystic fibrosis, has completed investigational new drug application-enabling activities [65]. Financial Performance - Research and development expenses for the three months ended March 31, 2020, totaled $5.289 million, compared to $4.197 million for the same period in 2019, reflecting an increase of approximately 26% [74]. - General and administrative expenses rose by $0.6 million to $2.8 million for the three months ended March 31, 2020, marking a 29% increase from the previous year [86]. - Total operating expenses for the three months ended March 31, 2020, were $8.0 million, up from $6.3 million in the same period of 2019, reflecting a $1.7 million increase [83]. - The net loss for the three months ended March 31, 2020, was $7.95 million, compared to a net loss of $6.04 million for the same period in 2019, an increase of $1.91 million [83]. - The company has an accumulated deficit of $231.5 million as of March 31, 2020, reflecting ongoing operating losses since inception [97]. Cash and Investments - As of March 31, 2020, the company had $55.1 million in cash, cash equivalents, and short-term investments, which are expected to support operations beyond a potential NDA filing into the third quarter of 2021 [98]. - Net cash used in operating activities was $7.0 million for the three months ended March 31, 2020, compared to $6.6 million for the same period in 2019 [92]. - Net cash provided by financing activities was $25.6 million during the three months ended March 31, 2020, primarily from net proceeds of $24.5 million from a public offering [95]. - The company raised an aggregate of $300.3 million from various financing activities since inception through March 31, 2020 [88]. - As of March 31, 2020, the company had cash, cash equivalents, and short-term investments totaling $55.1 million, an increase from $36.2 million as of December 31, 2019 [106]. - The company's cash equivalents as of March 31, 2020, included corporate debt securities and money market funds [106]. - Short-term investments as of March 31, 2020, consisted of corporate debt securities and U.S. reverse repurchase agreements [106]. Market Risk - The primary exposure to market risk is interest rate sensitivity, which is influenced by changes in U.S. interest rates [106]. - An immediate 10% change in interest rates would not have a material effect on the fair market value of the investment portfolio and interest income due to the low risk profile of the investments [106]. - As of March 31, 2020, the company had no material liabilities denominated in foreign currencies [107]. Research and Development Costs - The company anticipates significant increases in research and development costs due to the Phase 3 PolarisDMD trial and preparation for registration of edasalonexent [77]. - Total direct expenses for the edasalonexent program since inception amount to $52.6 million, while CAT-5571 has incurred $4.2 million [74]. - Research and development expenses increased by $1.1 million to $5.3 million for the three months ended March 31, 2020, representing a 26% increase compared to the same period in 2019 [84].